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Enrolling by invitation NCT04628585

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

— Observational Sickle Cell Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Safety and efficacy assessments.
Who it may be relevant to
Registry conditions: Sickle Cell Disease. Basic parameters: 2 years — 53 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, France
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector

Overview

This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies. After completing the parent clinical study (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in the study.

Interventions

  • Other Safety and efficacy assessments
    Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant

Primary outcome measures

  • Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV) [Time frame: Through 15 years post-drug product infusion]
  • Number of subjects with new or worsening hematologic disorders [Time frame: Through 15 years post-drug product infusion]
  • Number of subjects with new or worsening neurologic disorders [Time frame: Through 15 years post-drug product infusion]
  • Number of subjects with malignancies [Time frame: Through 15 years post-drug product infusion]
Secondary outcome measures (12)
  • Proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15 [Time frame: Through 15 years post-drug product infusion]
  • Proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15 [Time frame: Through 15 years post-drug product infusion]
  • Annualized number of severe VOEs over time through Year 15 [Time frame: Through 15 years post-drug product infusion]
  • Annualized number of VOEs over time through Year 15 [Time frame: Through 15 years post-drug product infusion]
  • Change from parent study baseline in annualized number of severe VOEs over time through Year 15 [Time frame: Through 15 years post-drug product infusion]
  • Assessment of total Hb over time post-drug product infusion through Year 15 [Time frame: Through 15 years post-drug product infusion]
  • Assessment of non-transfused total Hb over time post-drug product infusion through Year 15 [Time frame: Through 15 years post-drug product infusion]
  • Assessment of HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15 [Time frame: Through 15 years post-drug product infusion]
  • Assessment of HbAT87Q percentage of non-transfused total Hb over time post-drug product infusion through Year 15 [Time frame: Through 15 years post-drug product infusion]
  • Assessment of non-HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15 [Time frame: Through 15 years post-drug product infusion]
  • Change from parent study baseline through Year 15 in hemolysis markers [Time frame: Through 15 years post-drug product infusion]
  • Change from parent study baseline through Year 15 in markers of iron stores [Time frame: 15 years post-drug product infusion]

Eligibility criteria

Inclusion criteria

  • Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s)
  • Treated with drug product for therapy of sickle cell disease in a bluebird bio-sponsored clinical study

Exclusion criteria

  • There are no exclusion criteria for this study

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Case-only

Study locations

United States · 15 centers
  • University of Alabama — Birmingham
  • UCSF Benioff Children's Hospital Oakland — Oakland
  • Children's Healthcare of Atlanta — Atlanta
  • Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
  • Warren Grant Magnuson Clinical Center — Bethesda
  • University of Minnesota Masonic Children's Hospital — Minneapolis
  • Hackensack University Medical Center — Hackensack
  • Cohen Children's Medical Center — New Hyde Park
  • … and 7 more centers
France · 1 center
  • Hospital Necker — Paris

Publications

  • Magrin E, Semeraro M, Hebert N, Joseph L, Magnani A, Chalumeau A, Gabrion A, Roudaut C, Marouene J, Lefrere F, Diana JS, Denis A, Neven B, Funck-Brentano I, Negre O, Renolleau S, Brousse V, Kiger L, Touzot F, Poirot C, Bourget P, El Nemer W, Blanche S, Treluyer JM, Asmal M, Walls C, Beuzard Y, Schmidt M, Hacein-Bey-Abina S, Asnafi V, Guichard I, Poiree M, Monpoux F, Touraine P, Brouzes C, de Monta PMID 35075288

Identifiers

NCT: NCT04628585 · LTF-307 · 2019-004266-18 · 2024-513901-30-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗